Table 2 Summary of phase III clinical trials evaluating the efficacy of ASCT in the era of novel agent induction therapy

From: Utilization of hematopoietic stem cell transplantation for the treatment of multiple myeloma: a Mayo Stratification of Myeloma and Risk-Adapted Therapy (mSMART) consensus statement

Clinical trial

No.

Induction

Randomized arms

ORR

EFS/PFS

OS

Palumbo et al. [20]. NDMM pts <65 y/o

402

Rd × 4 cycles

MPR × 6 cycles →No maint

CR (post consolidation): 18%

Median PFS: 22 mos

5-year OS: 59%

MPR × 6 cycles →R-maint

 

Median PFS: 34 mos

5-year OS: 70%

Mel200 mg/m2 × 2 cycles → No maint

CR (post consolidation): 23%

Median PFS: 37 mos

5-year OS: 67%

Mel200 mg/m2 × 2 cycles → R-maint

 

Median PFS: 55 mos

5-year OS: 78%

Gay et al. [21]. NDMM pts ≤65 y/o

389

Rd × 4 cycles

CRd × 6 cycles → R-maint

CR: 27%

Median PFS: 28 mos

4-year OS: 76%

CRd × 6 cycles → Rp-maint

CR: 23%

Median PFS: 24 mos

4-year OS: 68%

Mel200 mg/m2 × 2 cycles → R-maint

CR: 33%

Median PFS: 32 mos

4-year OS: 75%

Mel200 mg/m2 × 2 cycles → Rp-maint

CR: 37%

Median PFS: 38 mos

4-year OS: 77%

Cavo et al. [23]. NDMM pts ≤65 y/o

1503

VCd × 3–4 cycles

VMP→+/− VRd → R-maint

≥VGPR: 75%

3-year PFS: 57%

Median OS: NR

Mel200 mg/m2 × 1 or 2 →+/− VRd →R-maint

≥VGPR: 84%

3-year PFS: 64%

Median OS: NR

Attal et al. [25]. NDMM pts ≤65 y/o

700

VRd × 3 cycles

VRd × 5 cycles →R-maint

CR: 48% MRD (−): 65%

Median PFS: 36 mos

4-year OS: 82%

Mel200 mg/m2 →VRd × 2 cycles →R-maint

CR: 59% MRD (−): 79%

Median PFS: 50 mos

4-year OS: 81%

  1. CRd cyclophosphamide, lenalidomide, and dexamethasone, R-maint lenalidomide maintenance, Rp-maint lenalidomide and prednisone maintenance, VRd bortezomib, lenalidomide, and dexamethasone, MPR melphalan, prednisone and lenalidomide, VCd bortezomib, cyclophosphamide and dexamethasone, ORR overall response rate, EFS event-free survival, PFS progression-free survival, OS overall survival, CR complete remission, MRD (−) minimal residual disease negativity